Feasibility in all settings Dr Kim Thomas Centre of Evidence Based - - PowerPoint PPT Presentation

Feasibility in all settings

Dr Kim Thomas Centre of Evidence Based Dermatology University of Nottingham

• Overview of some of the key issues
• Particular focus on “signs”, but applicable to all

domains

Overview

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• Interactive sessions and voting!

• What are we aiming for?

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Enough to get the job done?

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Types of trials

• Early-phase trials – high resource, frequent patient visits, often

require careful monitoring of interventions and potential side-effects.

• Phase IV, pragmatic - comparative effectiveness trials – does

the intervention work in “real life”? Long-term safety studies.

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• Large, multi-centre trials – multiple assessors and potentially

high turn-over of research staff.

• Self-funded trials – a clinician with a “good idea” and passion to

answer the question, could be a collaborative network of volunteer clinicians / nurses.

“Core outcome” that can be used in ALL trial settings

Pragmatic trials Open / unblinded trials Suitable for all severities & interventions

Need an “objective” scale Quick & easy to complete in a clinic setting Able to detect small differences?

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Long-term trials Low resource Patient- reported

• utcomes

Many investigators

Consistent & reliable over time Data management, data entry, postal follow-up? Training requirements, inter-observer variability Are we measuring things

• f importance to

patients, who completes the assessment?

BOTTOM LINE: is the scale sufficiently simple for it to be included even if that outcome is not

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even if that outcome is not relevant to the study in question?

Three item severity scale

• Assess THREE signs at a “representative” site

– Erythema (redness) – Excoriation (signs of scratching) – Oedema / papulation (swelling)

• These signs consistently been shown to associate

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• These signs consistently been shown to associate

with “worsening” of the disease

• Reasonably well validated, very quick and simple

Is it sufficient as a core outcome for eczema signs?

ANT Trial Trial of antihistamines versus no antihistamines in patients with moderate eczema

• 12 month, pragmatic trial
• Double-blind, participants seen in clinic at baseline

then followed-up by post / on-line questionnaire

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then followed-up by post / on-line questionnaire

• Primary outcome:

patient-assessed eczema severity assessed monthly by questionnaire (POEM scale?)

• Secondary outcomes: use of topical therapy, other

core outcomes for HOME (including eczema signs, long-term control and QoL)

PEST Trial Behavoural intervention for the management of moderate to severe eczema

• 6 month RCT – clinic visits every 2 months.
• Assessor blind

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• Primary outcome:

eczema severity – assessed by blinded research nurses

• Secondary outcomes: other core outcomes for

HOME (including eczema symptoms, long-term control and QoL)

What are we aiming for?

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Achieving “smart” core outcomes

“What is eczema?”

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“What improves as the eczema gets better?”

Focus on essential information

• What are we measuring and why?

Bleeding, blistering, cracks in the skin, crusting, scratch marks on the skin, involvement of sensitive / visible body sites, lichenification, redness, dryness, flaking,

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body sites, lichenification, redness, dryness, flaking, sleep difficulties, soreness or pain, swelling, amount of body affected, tightness of the skin, weeping / oozing

• Are all items necessary?

Are all items necessary?

• Some chronic signs less likely to change quickly

(e.g lichenification)

• Acute signs may be more sensitive to change

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– Redness (erythema) – Scratching (excoriation) – Swelling (oedema / papulation)

• Dryness (depends when emollient last applied, best

reported by patients?)

• Doctor-assessed itch…….

“doctor-assessed itch?”

• Should severity

assessment be made

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assessment be made by patients?

• Independent observers

can measure signs, but not symptoms.

Who assesses what?

• Our core domains currently focus on eczema signs

and symptoms as separate domains

• Should this be interpreted as:

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– Investigator-assessed severity (signs only) (suitable for unblinded studies) – Patient-assessed severity (symptoms & QoL)

Vote

• Add question

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• How are we measuring severity?

Severity at a “representative site” x “area of involvement”

• r

Severity assessed separately at multiple sites

• r

Focus on essential information

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• r

Global assessment

• Each has pros and cons

How representative is a “representative site”?

• What is a “representative site”?

An area of the body that represents: – a “typical” patch of eczema for the patient – a “typical” patch of eczema for a particular sign (e.g. signs of scratching) – the worst patch of eczema for the patient

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– the worst patch of eczema for the patient – the worst patch of eczema for a particular sign

• Do all body sites get worse / better at the same time?
• Are all sites equally important to patients?
• Is the same “representative” site used for subsequent

assessments?

• Discussion topic
• What does a “representative site” mean to you?

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Vote

• Add question

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Timeliness of the Core Set

• Do all domains need to

be ready at the same time?

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time?

Conclusion

• Put your preconceptions /

allegiances to one side

• Listen to all points of view

with an open mind

• Enjoy the discussion

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• Enjoy the discussion
• Be prepared to make

decisions

Disclaimer The HOME initiative is partially supported through an independent research programme funded by the National Institute for Health Research (NIHR) under its Programme Grants for Applied Research funding scheme (RP-PG-0407-10177). scheme (RP-PG-0407-10177). In particular, this grant has supported administration of the HOME project and patient representation at this HOME III meeting. The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health.

Combining data pre- and post- HOME

• Two of the most commonly used scales to date are:

– SCORAD (includes Three Item Severity scale) – EASI (includes Three Item Severity scale)

• Meta-analysis of old and new trials?

– How can we combine old and news trials to be in-line with

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core outcomes? – Could we encourage all to report TIS (3 signs) separately in all trials until final instrument has been decided?